Pulmozyme

Question

Hello,

Our son (6 years old, no modulators) is supposed to start Dornase Alpha. We would like to ask about your experience with its effectiveness?

Furthermore, despite a balanced intake of Kreon (pancreatic enzymes), he has a high calprotectin level (980). Do you have any suggestions regarding this?

Thank you very much for your reply.

Answer

Dear Sir/Madam,

Regarding Pulmozyme: it is approved for inhalation as a mucolytic agent for children aged 5 and older with cystic fibrosis; therefore, it is also suitable for your son. It is an "enzymatic" mucolytic agent that loosens mucus in a different way than the "hypertonic saline solution (6%)" that your son has likely been inhaling daily. The two medications complement each other. Studies have shown the best results when patients inhaled Pulmozyme once daily and also used hypertonic saline solution 1-2 times daily. I have many patients, especially children, for whom Pulmozyme was very effective, and for whom we even applied for its off-label use before the age of 5 when the hypertonic saline solution was insufficient for symptom control. Therefore, it shouldn't be inhaled directly before bedtime, as it often loosens so much mucus that it interferes with falling asleep or causes problems if the patient doesn't cough it up properly because they've already fallen asleep. So, definitely start with this in addition to the 6% saline solution.

Regarding calprotectin: it's an inflammatory marker measured in stool.

Normal calprotectin levels (may vary slightly from source to source):
below 50 µg/g stool is normal
between 50-200 µg/g stool is borderline elevated
over 200 µg/g stool is elevated

Elevated levels indicate an inflammatory response in the intestine, e.g., in chronic inflammatory bowel diseases.

In patients with cystic fibrosis (CF), calprotectin levels are indeed often higher than normal, as some inflammation in the intestine can occur in CF. Furthermore, there is data showing that it can also correlate with the severity of the disease in CF (Pediatr Gastroenterol Hepatol Nutr. 2022 Jan;25(1):1-12.
"Fecal Calprotectin and Phenotype Severity in Patients with Cystic Fibrosis: A Systematic Review and Meta-Analysis"; Saeedeh Talebi et al.)

In a 2019 medical doctoral thesis from the University of Giessen/Germany, researchers investigated calprotectin levels in patients with cystic fibrosis (CF) and in those without CF:

CF patients had higher calprotectin concentrations (mean 182 µg/g, range 66–363 µg/g) compared to non-CF patients (mean 17 µg/g, range 7–42 µg/g), but lower concentrations than patients with inflammatory bowel disease (mean 587 µg/g, range 270–1659 µg/g). 70% of CF patients had values ​​above 100 µg/g stool, and there was a trend towards higher calprotectin concentrations in patients with severe pulmonary dysfunction. 80% of CF patients had a thickened intestinal wall with significantly higher calprotectin levels. In CF patients, systemic inflammatory parameters correlated with stool calprotectin levels.

Thus, it becomes clear that a value of 980 The calprotectin level in stool (µg/g) needs further investigation, as elevated levels can occur in CF patients, but this level is unusual. Therefore, if the levels remain this high during follow-up examinations, an experienced pediatric gastroenterologist should be consulted. A colonoscopy may be necessary to rule out inflammatory bowel disease.

By the way, is it possible your son cannot receive modulators because he doesn't have a suitable mutation? This now only applies to about 10% of patients. If modulators are an option for him (Kaftrio/Trikafta is licensed for children aged 2 and above), he should use these medications, as they (partially) restore CFTR channel function and thus inhibit all inflammatory processes.

Definitely discuss this with your outpatient clinic!

I hope this has been helpful.

Sincerely,
Daniela d'Alquen

19.01.2026